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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Efficacy of deferasirox in reducing and preventing cardiac iron overload in β-thalassemia
Blood, Volume 115, No. 12, Year 2010
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Description
Cardiac iron overload causes most deaths in β-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with β-thalassemia in a 1-year prospective, multicenter study. The cardiac iron reduction arm (n = 114) included patients with magnetic resonance myocardial T2* from 5 to 20 ms (indicating cardiac siderosis), left ventricular ejection fraction (LVEF) of 56% or more, serum ferritin more than 2500 ng/mL, liver iron concentration more than 10 mg Fe/g dry weight, and more than 50 transfused blood units. The prevention arm (n = 78) included otherwise eligible patients whose myocardial T2* was 20 ms or more. The primary end point was the change in myocardial T2* at 1 year. In the cardiac iron reduction arm, the mean deferasirox dose was 32.6 mg/kg per day. Myocardial T2* (geometric mean ± coefficient of variation) improved from a baseline of 11.2 ms (± 40.5%) to 12.9 ms (± 49.5%) (+16%; P < .001). LVEF (mean ± SD) was unchanged: 67.4 (± 5.7%) to 67.0 (± 6.0%) (-0.3%; P = .53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (± 25.6%) to 32.5 ms (± 25.1%) (±2%; P = .57) and LVEF increased from baseline 67.7 (± 4.7%) to 69.6 (± 4.5%) (+1.8%; P < .001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation. This study is registered at http:// clinicaltrials.gov as NCT00171821. © 2010 by The American Society of Hematology.
Authors & Co-Authors
Pennell, Dudley John
United Kingdom, London
Royal Brompton Hospital
Porter, John B.
United Kingdom, London
University College London
Cappellini, Maria Teresa
Italy, Milan
Università Degli Studi Di Milano
El-Beshlawy, Amal M.
Egypt, Giza
Cairo University
Chan, Lee Lee
Malaysia, Kuala Lumpur
University of Malaya Medical Centre
Aydınok, Yeşim
Turkey, Izmir
Ege University Medical School
ElAlfy, Mohsen Saleh
Egypt, Cairo
Ain Shams University
Sutcharitchan, Pranee
Thailand, Bangkok
Chulalongkorn University
Li, Chi Kong
Hong Kong
Prince of Wales Hospital Hong Kong
Ibrahim, Hishamshah
Malaysia, Kuala Lumpur
Kuala Lumpur Hospital
Viprakasit, Vip
Thailand, Bangkok
Faculty of Medicine Siriraj Hospital, Mahidol University
Kattamis, Antonis C.
Greece, Athens
National and Kapodistrian University of Athens
Smith, Gillian C.
United Kingdom, London
Royal Brompton Hospital
Habr, Dany
Switzerland, Basel
Novartis International ag
Domokos, Gabor
Switzerland, Basel
Novartis International ag
Roubert, Bernard
Switzerland, Basel
Novartis International ag
Taher, Ali T.
Lebanon, Beirut
American University of Beirut
Statistics
Citations: 199
Authors: 17
Affiliations: 14
Identifiers
Doi:
10.1182/blood-2009-04-217455
ISSN:
00064971
Research Areas
Noncommunicable Diseases
Study Design
Cohort Study